Abstract Details

Title Juvenile Myasthenia Gravis: Evaluation of presentation and treatment outcomes in a large cohort

Topic Child Neurology and Developmental Neurology

Presentation(s) P5 - Poster Session 5 (5:30 PM-6:30 PM)

Poster/Presentation
Number 7-070

Objective To investigate clinical presentation, course, and response to treatment of myasthenia gravis in pediatric patients in a large single center cohort.

Background Myasthenia gravis is an autoimmune disease in which autoantibodies target the neuromuscular junction. In adults, well delineated symptoms of muscle weakness and fatigability are disease hallmarks. Clinical presentation and diagnosis in pediatric populations can be elusive. These patients have increased risk for other autoimmune diseases, have less well defined auto-antibody associations, and have different clinical characteristics. The management and outcomes vary broadly, especially with new immunosuppressive modalities becoming available.

Design/Methods

Retrospective chart review of electronic medical records of all children with ICD9/10 diagnosis of myasthenia gravis presenting to Texas Children's Hospital.

Results

The average age at presentation in this cohort was 8.2 years. Four patients (8%) had congenital myasthenia, while forty six (92%) had presumed autoimmune myasthenia, all of whom were tested for causative antibodies (acetylcholine receptor or muscle specific kinase). Of the tested patients, twenty four (48%) tested positive. Twenty two (44%) continued on an immunosuppressive agent. Four (8%) had a separate autoimmune disease, of which two were autoimmune thyroiditis and two were type I diabetes. The most common exam finding was ptosis. Ptosis was found at some point in forty eight patients (96%) during their care. In terms of treatment, nine (18%) of our patients had thymectomy. 78% of the patients had good outcomes, of which patients who were on medical management only had better rates of good outcomes (85%) as compared to medical and surgical management (44%).

Conclusions The present study offers one of the largest cohorts of pediatric MG patients who have undergone up to date diagnostic and therapeutic regimens. The responses to therapy in this pediatric cohort will help to guide future treatment of pediatric myasthenia gravis.

Authors/Disclosures
Jason S. Gill, MD, PhD (Baylor College of Medicine) PRESENTER	Dr. Gill has received personal compensation in the range of $500-$4,999 for serving as an Expert Witness for Elite Medical Experts. The institution of Dr. Gill has received research support from NIH-NINDS.
	No disclosure on file
Timothy E. Lotze, MD, FAAN (Texas Children's Hospital)	Dr. Lotze has received personal compensation in the range of $5,000-$9,999 for serving as an Expert Witness for Department of Justice VICP. The institution of Dr. Lotze has received research support from NIH. The institution of Dr. Lotze has received research support from National MS Society. The institution of Dr. Lotze has received research support from Sarepta Therapeutics. The institution of Dr. Lotze has received research support from PTC THERAPEUTICS. The institution of Dr. Lotze has received research support from Avexis. Dr. Lotze has received publishing royalties from a publication relating to health care. Dr. Lotze has received publishing royalties from a publication relating to health care.

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